Outcome of high-risk stage 3 neuroblastoma with myeloablative therapy and 13-cis-retinoic acid: a report from the Children's Oncology Group.
BACKGROUND
The components of therapy required for patients with INSS Stage 3 neuroblastoma and high-risk features remain controversial.


METHODS
A retrospective cohort design was used to determine if intensive chemoradiotherapy with purged autologous bone marrow rescue (ABMT) and/or 13-cis-retinoic acid (13-cis-RA) improved outcome for patients with high-risk neuroblastoma that was not metastatic to distant sites. We identified 72 patients with INSS Stage 3 neuroblastoma enrolled between 1991 and 1996 on the Phase 3 CCG-3891 randomized trial. Patients were analyzed on an intent-to-treat basis using a log-rank test.


RESULTS
The 5-year event-free survival (EFS) and overall survival (OS) rates for patients with Stage 3 neuroblastoma were 55 +/- 6% and 59 +/- 6%, respectively (n = 72). Patients randomized to ABMT (n = 20) had 5-year EFS of 65 +/- 11% and OS of 65 +/- 11% compared to 41 +/- 11 (P = 0.21) and 46 +/- 11% (P = 0.23) for patients randomized to CC (n = 23), respectively. Patients randomized to 13-cis-RA (n = 23) had 5-year EFS of 70 +/- 10% and OS of 78 +/- 9% compared to 63 +/- 12% (P = 0.67) and 67 +/- 12% (P = 0.55) for those receiving no further therapy (n = 16), respectively. Patients randomized to both ABMT and 13-cis-RA (n = 6) had a 5-year EFS of 80 +/- 11% and OS of 100%.


CONCLUSIONS
Patients with high-risk Stage 3 neuroblastoma have an overall poor prognosis despite aggressive chemoradiotherapy. Further studies are warranted to determine if myeloablative consolidation followed by 13-cis-RA maintenance therapy statistically significantly improves outcome.
Park
Julie R
JR
Department of Pediatrics, Children's Hospital and Regional Medical Center, University of Washington, Seattle, Washington 98105, USA. julie.park@seattlechildrens.org
Villablanca
Judith G
JG
London
Wendy B
WB
Gerbing
Robert B
RB
Haas-Kogan
Daphne
D
Adkins
E Stanton
ES
Attiyeh
Edward F
EF
Maris
John M
JM
Seeger
Robert C
RC
Reynolds
C Patrick
CP
Matthay
Katherine K
KK
D016465:Bone Marrow Purging; D016026:Bone Marrow Transplantation / Q000379:methods*; D002945:Cisplatin; D003131:Combined Modality Therapy; D003520:Cyclophosphamide; D004317:Doxorubicin; D005047:Etoposide; D006801:Humans; D007223:Infant; D015474:Isotretinoin / Q000627:therapeutic use*; D009447:Neuroblastoma / Q000401:mortality / Q000628:therapy*; D011878:Radiotherapy; D012306:Risk; D016019:Survival Analysis; D014182:Transplantation, Autologous; D016896:Treatment Outcome
D005047:Etoposide; D004317:Doxorubicin; D003520:Cyclophosphamide; D015474:Isotretinoin; D002945:Cisplatin